A new drug is extending both remission and survival in adults with acute myeloid leukemia (AML), according to findings reported at the American Society of Hematology annual meeting in December 2019. The study was funded by Celgene, the drug’s manufacturer.

CCC-486 is an investigational treatment similar to azacitidine, but it’s an oral therapy that could be taken at home instead of an IV or injection at the cancer clinic.

For the international trial, researchers enrolled 472 patients with AML aged 55 or older within four months of experiencing remission with initial chemotherapy treatment. Participants were randomly assigned to receive either CC-486 or a placebo, and treatment continued until patients relapsed or developed unacceptable side effects.

Median survival was 25 and 15 months and relapse-free survival was 10 and 5 months in the CC-486 and placebo groups, respectively. The researchers reported that the survival benefits existed regardless of patients’ original risk for relapse, the number of consolidation chemotherapy cycles they received, or whether they were in complete remission versus complete remission with incomplete count recovery.

Common side effects included nausea, vomiting, and diarrhea. More patients in the CC-486 group experienced serious side effects, including neutropenia and infections. The few patients who chose to discontinue treatment did so because of gastrointestinal side effects.

“CC-486 is the first therapy to provide statistically significant and clinically meaningful improvement in both overall survival and relapse-free survival, with or without consolidation,” the researchers concluded.